Processing

Please wait...

Settings

Settings

1. WO2020006267 - COMPOUNDS AND METHODS FOR REDUCING LRRK2 EXPRESSION

Publication Number WO/2020/006267
Publication Date 02.01.2020
International Application No. PCT/US2019/039558
International Filing Date 27.06.2019
IPC
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
7115
Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
712
Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
7125
Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
C12N 15/113 (2010.01)
A61K 31/7115 (2006.01)
A61K 31/712 (2006.01)
A61K 31/7125 (2006.01)
CPC
A61K 31/7115
A61K 31/712
A61K 31/7125
C12N 15/1137
C12N 2310/11
C12N 2310/315
Applicants
  • IONIS PHARMACEUTICALS, INC. [US/US]; 2855 Gazelle Court Carlsbad, CA 92010, US
Inventors
  • COLE, Tracy, A.; US
  • KORDASIEWICZ, Holly; US
  • BUI, Huynh-Hoa; US
  • FREIER, Susan, M.; US
Agents
  • SCARR, Rebecca, B.; US
  • MCNEILL, Rebecca; US
  • KELLEY, Lara; US
  • DOHERTY, Eilizabeth; US
  • HERZFELD, Deborah, M.; US
Priority Data
62/690,79027.06.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOUNDS AND METHODS FOR REDUCING LRRK2 EXPRESSION
(FR) COMPOSÉS ET PROCÉDÉS PERMETTANT DE RÉDUIRE L'EXPRESSION DE LRRK2
Abstract
(EN)
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least 5 one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.
(FR)
L'invention concerne des composés, des procédés et des compositions pharmaceutiques permettant de réduire la quantité ou l'activité de l'ARN LRRK2 dans une cellule ou chez un animal et, dans certains cas, de réduire la quantité de LRRK2 dans une cellule ou chez un animal. Des tels composés, procédés et compositions pharmaceutiques sont utiles pour atténuer au moins un symptôme ou un signe d'une maladie neurodégénérative. Ces symptômes et ces signes comprennent l'ataxie, la neuropathie et la formation d'agrégats. De telles maladies neurodégénératives comprennent la maladie de Parkinson.
Latest bibliographic data on file with the International Bureau